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Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides
Karima Relizani
,
Lucía Echevarría
,
Faouzi Zarrouki
,
Cécile Gastaldi
,
Chloe Dambrune
,
et al.
Article dans une revue
hal-03476811v1
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Exon Skipping in a Dysf-Missense Mutant Mouse Model
Jakub Malcher
,
Leonie Heidt
,
Aurélie Goyenvalle
,
Helena Escobar
,
Andreas Marg
,
et al.
Article dans une revue
hal-02407941v1
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Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment
Valérie Robin
,
Graziella Griffith
,
John-Paul Carter
,
Christian Leumann
,
Luis Garcia
,
et al.
Article dans une revue
hal-02407957v1
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RNA-targeted drugs for neuromuscular diseases
Alessandra Ferlini
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Aurélie Goyenvalle
,
Francesco M. Muntoni
Article dans une revue
hal-03127509v1
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Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
Philippine Aupy
,
Faouzi Zarrouki
,
Quentin Sandro
,
Cécile Gastaldi
,
Pierre-Olivier Buclez
,
et al.
Article dans une revue
hal-02912922v1
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Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model
Lucía Echevarría
,
Philippine Aupy
,
Karima Relizani
,
Thomas Bestetti
,
Graziella Griffith
,
et al.
Article dans une revue
hal-02407933v1
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Un nouvel outil pour le traitement de la myopathie de Duchenne : les tricyclo-ADN
Aurélie Goyenvalle
,
Graziella Griffith
,
Aurélie Avril
,
Helge Amthor
,
Luis Garcia
Article dans une revue
hal-02407963v1
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Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models
Amel Saoudi
,
Claire Fergus
,
Talia Gileadi
,
Federica Montanaro
,
Jennifer E. Morgan
,
et al.
Article dans une revue
hal-04068549v1
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Les approches thérapeutiques de modulation de l’épissage
Amel Saoudi
,
Aurélie Goyenvalle
Article dans une revue
hal-03273143v1
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Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model
Karima Relizani
,
Graziella Griffith
,
Lucía Echevarría
,
Faouzi Zarrouki
,
Patricia Facchinetti
,
et al.
Article dans une revue
hal-02407950v1
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Altered visual processing in the mdx52 mouse model of Duchenne muscular dystrophy
Mirella Telles Salgueiro Barboni
,
André Maurício Passos Liber
,
Anneka Joachimsthaler
,
Amel Saoudi
,
Aurelie Goyenvalle
,
et al.
Article dans une revue
hal-03196031v1
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uAUG creating variants in the 5’UTR of ENG causing Hereditary Hemorrhagic Telangiectasia
Omar Soukarieh
,
Emmanuelle Tillet
,
Carole Proust
,
Charlène Dupont
,
Béatrice Jaspard-Vinassa
,
et al.
Article dans une revue
hal-04271267v1
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Live-imaging of revertant and therapeutically restored dystrophin in the DmdEGFP-mdx mouse model for Duchenne muscular dystrophy
Mina V. Petkova
,
Amalia Stantzou
,
A. Morin
,
O. Petrova
,
Susanne Morales-Gonzalez
,
et al.
Article dans une revue
hal-02984274v2
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Rescue of a dystrophin-like protein by exon skipping in vivo restores GABAA-receptor clustering in the hippocampus of the mdx mouse.
Cyrille Vaillend
,
Caroline Perronnet
,
Carine Ros
,
Carole Gruszczynski
,
Aurélie Goyenvalle
,
et al.
Article dans une revue
hal-01183355v1
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Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy
Philippine Aupy
,
Lucía Echevarría
,
Karima Relizani
,
Faouzi Zarrouki
,
Adrian Haeberli
,
et al.
Article dans une revue
hal-02530970v1
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Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping
Saleh Omairi
,
Kwan-Leong Hau
,
Henry Collin-Hooper
,
Federica Montanaro
,
Aurélie Goyenvalle
,
et al.
Article dans une revue
hal-02407948v1
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Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
Simon Krooss
,
Sonja Werwitzke
,
Johannes Kopp
,
Alice Rovai
,
Dirk Varnholt
,
et al.
Article dans une revue
inserm-02877214v1
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Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides
Aurélie Goyenvalle
,
Cecilia Jimenez-Mallebrera
,
Willeke M.C. van Roon
,
Sabine Alex Sewing
,
Arthur M. Krieg
,
et al.
Article dans une revue
hal-04068676v1
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Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers
Aurélie Goyenvalle
,
Graziella Griffith
,
Arran Babbs
,
Samir El Andaloussi
,
Kariem Ezzat
,
et al.
Article dans une revue
hal-02407968v1
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Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
Flavien Bizot
,
Remko Goossens
,
Thomas Tensorer
,
Sergei Dmitriev
,
Luis Garcia
,
et al.
Article dans une revue
hal-03891253v1
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Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X‐Linked ( mdx ) Mouse
Faouzi Zarrouki
,
Karima Relizani
,
Flavien Bizot
,
Thomas Tensorer
,
Luis Garcia
,
et al.
Article dans une revue
hal-03713396v1
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Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders
Flavien Bizot
,
Adeline Vulin
,
Aurélie Goyenvalle
Article dans une revue
hal-02912701v1
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Abnormal Expression of Synaptic and Extrasynaptic GABAA Receptor Subunits in the Dystrophin-Deficient mdx Mouse
Faouzi Zarrouki
,
Sébastien Goutal
,
Ophélie Vacca
,
Luis Garcia
,
Nicolas Tournier
,
et al.
Article dans une revue
hal-03839801v1
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Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model
Flavien Bizot
,
Abdallah Fayssoil
,
Cécile Gastaldi
,
Tabitha Irawan
,
Xaysongkhame Phongsavanh
,
et al.
Article dans une revue
hal-04021748v1
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Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model
Mathilde Doisy
,
Ophélie Vacca
,
Claire Fergus
,
Talia Gileadi
,
Minou Verhaeg
,
et al.
Article dans une revue
hal-04491701v1
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Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice
Amel Saoudi
,
Sacha Barberat
,
Olivier Le Coz
,
Ophélie Vacca
,
Mathilde Doisy Caquant
,
et al.
Article dans une revue
hal-04106304v1
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Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle
A. Morin
,
Amalia Stantzou
,
Olga N. Petrova
,
John C.W. Hildyard
,
T. Tensorer
,
et al.
Article dans une revue
hal-04122777v1
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Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle
Adrien Morin
,
Amalia Stantzou
,
Olga Petrova
,
John Hildyard
,
Thomas Tensorer
,
et al.
Article dans une revue
hal-04302715v1
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Impaired Adaptive Response to Mechanical Overloading in Dystrophic Skeletal Muscle
Pierre Joanne
,
Christophe Hourde
,
Julien Ochala
,
Yvain Cauderan
,
Fadia Medja
,
et al.
Article dans une revue
hal-01545458v1
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